Rocket Pharmaceuticals Reports Full Year 2017 Financial Results and Operational Highlights

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today reported financial results and operational highlights for the full year ended December 31, 2017.

“2017 was an important year for Rocket. We advanced our pipeline of gene therapy programs focused on life-threatening and devastating pediatric diseases, and we implemented strategic initiatives that have strengthened our organization,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “We believe Rocket’s multi-platform development approach uniquely positions us to aggressively pursue a broad range of best-in-class indications, enabling first-mover advantage.”

“With the successful closing of our merger with Inotek Pharmaceuticals and recent public offering in January, we enter 2018 with a solid financial position to accomplish our key clinical and operational goals,” continued Dr. Shah. “In addition to more clinical data on our lead program in Fanconi Anemia (FA), we plan to announce the disease indication for our adeno-associated viral vector (AAV) gene therapy program in the second half of this year. We also expect to file Investigational Medicinal Product Dossier (IMPD) applications for our Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) programs, as well as an Investigational New Drug (IND) application for our AAV program over the next 12 months. With multiple programs moving forward in parallel, we believe we have the key elements in place to build a transformative, multi-platform gene therapy company delivering sustained, long-term growth for our shareholders.”

Recent Pipeline and Corporate Updates

• Promising preliminary patient data reported for RP-L102, a Phase 1/2 lentiviral vector (LVV)-based gene therapy for FA. At the European Society of Gene and Cell Therapy’s Annual Meeting in October 2017, Rocket’s collaboration partners at the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain presented interim results from the ongoing Phase 1/2 trial of RP-L102 in FA. Data presented in FA patients, without the use of conditioning, showed promising in vivo engraftment and stabilization of blood counts despite declining counts in the months to years preceding therapy.
• Studies of FA gene therapy product, RP-L101, utilizing new gene modified cell product manufacturing process underway. At the American Society of Hematology Annual Meeting in December 2017, Rocket’s collaboration partners at the Fred Hutchinson Cancer Research Center presented data from a pilot clinical trial assessing the feasibility of a novel lineage depletion protocol for hematopoietic stem cell (HSC) enrichment and subsequent transduction with the LVV-based gene therapy, RP-L101. Collection, lentiviral transduction and re-infusion of gene modified cells was shown to effectively produce long-term engraftment in mice. Patient treatment with this novel protocol is underway.
• Three additional LVV-based gene therapy pipeline programs in rare bone marrow disorders advance towards the clinic. LAD-I and PKD programs continue to move forward in preclinical IND-enabling studies. An additional program targeting Infantile Malignant Osteopetrosis (IMO) is also in preclinical development. Similar to FA, these diseases can lead to serious complications and have very limited long-term safe and effective treatment options.
• Rocket commenced trading on the NASDAQ Global Market under the symbol “RCKT”, following the successful closing of its with merger with Inotek Pharmaceuticals Corporation (Inotek) in January. In conjunction with the completion of the merger, the Company announced its executive management team will be led by Dr. Shah and will consist of: Jonathan Schwartz, M.D., Chief Medical Officer, and Kinnari Patel, Pharm.D., MBA, Chief Operating Officer.
• Rocket completed an oversubscribed underwritten public offering of 6,325,000 shares of its common stock in January 2018. Net proceeds to Rocket from the offering were approximately $78.8 million after deducting underwriting discounts and commissions.

Anticipated Milestones